Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Phase 3 Completed 85 enrolled Mirum Pharmaceuticals, Inc. Children's Hospital Medical Center, Cincinnati · collab

NaSingle-groupOpen-labelTreatment

Summary

OBJECTIVES: I. To Evaluate the therapeutic efficacy of cholic acid during provision of compassionate treatment to patients with identified inborn errors of bile acid synthesis and metabolism II. To assess the safety and tolerability of cholic acid

Timeline

Start: 1992-01
Primary completion: 2009-12
Completion: 2009-12

Publications

Heubi JE, Bove KE, Setchell KDR. Oral Cholic Acid Is Efficacious and Well Tolerated in Patients With Bile Acid Synthesis and Zellweger Spectrum Disorders. J Pediatr Gastroenterol Nutr. 2017 Sep;65(3):321-326. doi: 10.1097/MPG.0000000000001657.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	Cholic Acid	Small molecule	10 mg/kg	Oral
Subject	Cholic Acid	Small molecule	15 mg/kg	Oral

Compassionate Treatment of Patients With Inborn Errors of Bile Acid Metabolism With Cholic Acid

Summary

Timeline

Publications

Drugs

Indications